Evidence map›Paper›PMID 32223895›Full record

ArticleeLife2020

Identification of drug modifiers for RYR1-related myopathy using a multi-species discovery pipeline.

Jonathan R Volpatti, Yukari Endo, Jessica Knox, Linda Groom, Stephanie Brennan, Ramil Noche, William J Zuercher, Peter Roy, Robert T Dirksen, James J Dowling

Open access · goldAbstract read
In one paragraph

Article in eLife, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
1.9field-weighted citation impact, top 16% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 23 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Unconventional Myosins fromBiomolecules · 2022
    Review
  5. Article
  6. Review
  7. Article
  8. Article
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  11. Article
  12. Review
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  18. Zebrafish Models ofFrontiers in molecular neuroscience · 2020
    Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 4 institutions in 2 countries.

Jonathan R VolpattiProgram for Genetics and Genome Biology, Hospital for Sick Children, Toronto, Canada.
Yukari EndoProgram for Genetics and Genome Biology, Hospital for Sick Children, Toronto, Canada.
Jessica KnoxDepartment of Molecular Genetics, University of Toronto, Toronto, Canada.
Linda GroomDepartment of Pharmacology, University of Rochester, Rochester, United States.
Stephanie BrennanProgram for Genetics and Genome Biology, Hospital for Sick Children, Toronto, Canada.
Ramil NocheProgram for Genetics and Genome Biology, Hospital for Sick Children, Toronto, Canada.
William J ZuercherUNC Eshelman School of Pharmacy, SGC Center for Chemical Biology, University of North Carolina, Chapel Hill, United States.
Peter RoyDepartment of Molecular Genetics, University of Toronto, Toronto, Canada.
Robert T DirksenDepartment of Pharmacology, University of Rochester, Rochester, United States.ORCID 0000-0002-3182-1755
James J DowlingProgram for Genetics and Genome Biology, Hospital for Sick Children, Toronto, Canada.ORCID 0000-0002-3984-4169
University of Toronto · CAHospital for Sick Children · CAUniversity of Rochester · USUniversity of North Carolina at Chapel Hill · US

Funding

CIHR 363863
6 · The paper itself

Abstract

Ryanodine receptor type I-related myopathies (RYR1-RMs) are a common group of childhood muscle diseases associated with severe disabilities and early mortality for which there are no available treatments. The goal of this study is to identify new therapeutic targets for RYR1-RMs. To accomplish this, we developed a discovery pipeline using nematode, zebrafish, and mammalian cell models. We first performed large-scale drug screens in

Indexed as

AnimalsCaenorhabditis elegansCalciumDrug DiscoveryGene Knockout TechniquesHigh-Throughput Screening AssaysMiceMuscle Fibers, SkeletalMuscular DiseasesNeuromuscular Diseasesp38 Mitogen-Activated Protein KinasesPharmaceutical PreparationsRNA InterferenceRyanodine Receptor Calcium Release ChannelSmall Molecule LibrariesZebrafishCalciump38 Mitogen-Activated Protein KinasesPharmaceutical PreparationsRyanodine Receptor Calcium Release ChannelSmall Molecule Librariesanimal modelsC. elegansdrug screenexcitation contraction couplinghuman biologymedicinemousemyopathyRYR1zebrafish

Identifiers

PMID32223895
PMCPMC7202896
OpenAlexW3013089206

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.